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Published in May 2010NAMED PATIENT ProgramsNamed Patient ProgramsProvide Pre-Launch Access to DrugsThe dire outlook facing AML patients motivated a company to provide its drug in advance of the commercial launch.Contributed by
Named patient programs (NPPs) enable physi-
cians and patients in Europe to access medications
JACK V. TALLEYPRESIDENT AND CEO, EPICEPT
Medicines Agency (EMEA), but not yet launchedcommercially in European Union (EU) member
EpiCept is a specialty pharmaceutical company
countries. Regulations also allow patients to access
that focuses on fulfilling unmet medical needs
drugs that are approved outside of the EU, but not
in cancer treatment and pain management.
yet in their home countries. In both scenarios,requests for access are made by physicians on behalf
product and a deep and balanced pipeline of
Ceplene is available on a named patient basis in
three major clinical product candidates. For
Europe and in dozens of other key markets, including
more information, visit epicept.com or e-mail
Latin America, Asia/Pacific, Australia, Israel, and Canada.
Similar programs that enable pre-approval or pre-
launch access exist in many countries around theworld; respective governments have set well-definedrules for access criteria, collection of safety data, and
The grim prospects for long-term survival in acute
control of drug distribution. In the United States, such
myeloid leukemia (AML) strongly motivated EpiCept
government-sanctioned initiatives are referred to as
to provide patients a route of access in advance of
expanded access programs and may be implement-
the commercial introduction of its drug, Ceplene, the
ed for individuals, intermediate-sized groups (10 to
first approved immunotherapy for remission mainte-
100 patients), and large groups of patients.
nance and prevention of relapse in adult AMLpatients in first remission.THE ROUTE TO ACCESS
At the heart of the decision to establish a pre-
Time Frame for Planning a Named Patient Program
launch or pre-approval access program is the patientwith an unmet medical need. EpiCept sought to offer
a route to access that was legal and ethical. Fuelinginterest in providing access were numerous requests
being received by the company from physicians in
Europe and other parts of the world.
The successful development and implementa-
tion of NPPs require the involvement and coordina-
tion of many disciplines within the sponsoring com-pany, including representatives from medical affairs,pharmaceutical development, regulatory, supply
chain, business development, and finance. The cross-
functional team is responsible for ensuring that theclinical criteria for patient participation are estab-lished, physician educational materials are defined,the supply of drug is adequate to support the pro-gram, and enrollment in any ongoing clinical trials
6 MONTHS 12 MONTHS
The NPP has allowed EpiCept to make Ceplene
Note: The optimal time frame for implementing the program is about 12 months
available to patients in the following situations:
before the drug is expected to receive approval.
• In the EU following centralized approval but prior
to commercial launch in member countries.PharmaVOICENAMED PATIENT ProgramsNamed Patient Program Requirements in Select EU Member StatesLimited to ProductsLicensed AuthorizationApplication toPrescriptionFol ow-upElsewhere?Necessary?Competent Authority?ApplicantRequired?LiabilityWho Pays?Obligations
• In countries where applications for approval were
As with all NPPs, the sponsoring company has a
cal need who do not meet a clinical trial’s inclusion
responsibility to report serious adverse reactions to
• In countries where approval won’t be sought.
• Bridging the gap between the end of Phase III and
While the drug approval process in Europe is
Working with Idis, EpiCept established a full phar-
centralized via the EMEA, the drug reimbursement
macovigilance system to capture and report such
• Bridging the gap between approval and commer-
system is decentralized; decisions regarding reim-
information. The company also established a series of
bursement take place within each respective EU
standard operating procedures to guide the pro-
• Throughout a staggered global launch while
country. This process often results in a delay in com-
gram — these include procedures related to enroll-
approval and reimbursement is being sought
mercial launch as some countries can take between
ment of patients, release of the drug to the hospital,
12 and 18 months to establish reimbursement fol-
as well as regular reporting mechanisms on usage.
For patients with life-threatening illnesses such as
lowing EMEA approval of a drug. During this lag peri-
Programs in the various countries will terminate
AML, license approval or commercial launch of an
od, AML patients in the EU can access Ceplene
with the commercial launch of the drug. Those
innovative new drug in their home country may
patients accessing Ceplene on a named patient
come too late. In some cases — those involving the
While pre-launch access is permitted by EU legis-
basis will simply transition to the commercial drug
EU registration and approval process, for example —
lation, NPPs are governed by the individual member
once available and continue treatment in an unin-
a drug may not clear reimbursement hurdles in an
states; each of the 30 member states of the European
terrupted manner. In countries where approval is not
individual country until well after centralized
Economic Area (EEA) has its own regulations regard-
being sought, patients will continue to access Cep-
approval is granted. In cases where a formal launch
may not even be planned, a patient may not have
To navigate the regulatory and logistical path-
any other opportunity to receive the drug other than
ways in more than 100 countries and remain in com-
MEETING UNMET NEEDS
pliance with all authorities, EpiCept chose to partner
These programs also provide access to patients
with Idis, a specialist in NPP development and man-
Pre-launch access to drugs can provide profound
who would otherwise not be able to participate in
agement, rather than rely on internal resources.
benefits for patients with unmet medical needs
company-sponsored clinical trials and allow spon-
Once the decision was made to offer Ceplene on
when all other therapeutic options have failed or no
soring companies to engage with physicians and
a named patient basis, establishment of the program
other options are available. NPPs facilitate patient
provide early, hands-on access to life-saving
required about 10 weeks. Initiation of the NPP in the
access in markets that a company may not be pur-
medicines. In all of these scenarios, named patient
EU was timed to coincide with the date the drug was
suing in any other way. For example, it can be imprac-
programs allow companies to provide access to
ready from a manufacturing standpoint and had
tical for a small company to credibly pursue approval
innovative drugs in an ethical, legal manner while
complete and final EMEA approval and sign off.
in more than 100 countries, such as those that the
maintaining strict control over where the drugs go
Along with each request for Ceplene the pre-
scribing physician provides the patient’s age and
Using NPPs, access can be expanded to markets
remission status. EpiCept provides complete pre-
that would otherwise not be served, where gaps
scribing information as contained within the Sum-
exist between approval and launch or in situations
article. E-mail us at [email protected]
mary of Product Characteristics (SmPC) to those
where a patient cannot enter a clinical trial.
physicians requesting the drug. A copy of a peer-
Named patient programs can provide access at a
To download a FREE Podcast from Idis on Named Patient
reviewed article on Ceplene’s Phase III clinical trial is
number of stages throughout a product’s life cycle:
Programs, visit www.pharmavoice.com/podcasts
• During Phase III, for patients with an unmet medi-
PharmaVOICE